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Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Author

Listed:
  • Mark E. Davis

    (Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA)

  • Jonathan E. Zuckerman

    (Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA)

  • Chung Hang J. Choi

    (Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA)

  • David Seligson

    (Jonsson Comprehensive Cancer Center,
    David Geffen School of Medicine)

  • Anthony Tolcher

    (START - South Texas Accelerated Research Therapeutics, LLC, 4383 Medical Drive, 4th Floor, San Antonio, Texas 78229, USA)

  • Christopher A. Alabi

    (Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA
    Present address: Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.)

  • Yun Yen

    (City of Hope Comprehensive Cancer Center, 1500 E. Duarte Road, Duarte, California 91010, USA)

  • Jeremy D. Heidel

    (Calando Pharmaceuticals, 201 South Lake Avenue, Suite 703, Pasadena, California 91101, USA)

  • Antoni Ribas

    (Jonsson Comprehensive Cancer Center,
    David Geffen School of Medicine, University of California, Los Angeles, California 90095, USA)

Abstract

Human RNAi therapy The ability to downregulate specific genes using systemically delivered short RNA molecules and the cellular mechanism known as RNA interference has been shown previously in mouse and non-human primate models. Davis et al. have now demonstrated for the first time in humans that a short interfering RNA (siRNA) molecule can be systemically delivered using nanoparticles to a solid tumour. The siRNA mediates directed cleavage of its target mRNA, thereby also reducing the protein level. This proof-of-principle study confirms the potential of this technology as a human therapeutic.

Suggested Citation

  • Mark E. Davis & Jonathan E. Zuckerman & Chung Hang J. Choi & David Seligson & Anthony Tolcher & Christopher A. Alabi & Yun Yen & Jeremy D. Heidel & Antoni Ribas, 2010. "Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles," Nature, Nature, vol. 464(7291), pages 1067-1070, April.
    • Handle: RePEc:nat:nature:v:464:y:2010:i:7291:d:10.1038_nature08956
    DOI: 10.1038/nature08956
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    Cited by:

    1. A. Kuznetsov, 2012. "Modelling transport of layered double hydroxide nanoparticles in axons and dendrites of cortical neurons," Computer Methods in Biomechanics and Biomedical Engineering, Taylor & Francis Journals, vol. 15(12), pages 1263-1271.
    2. Tomoko Nakanishi & Julian Willett & Yossi Farjoun & Richard J. Allen & Beatriz Guillen-Guio & Darin Adra & Sirui Zhou & J. Brent Richards, 2023. "Alternative splicing in lung influences COVID-19 severity and respiratory diseases," Nature Communications, Nature, vol. 14(1), pages 1-12, December.

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